Title : Consistently High Agreement Between Independent Raters of Niemann-Pick Type C1 Clinical Severity Scale in Phase 2/3 Trial.

Pub. Date : 2022 Feb

PMID : 34952292






1 Functional Relationships(s)
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1 BACKGROUND: Niemann-Pick disease, type C1 (NPC1) is a rare neurodegenerative genetic disorder characterized by impaired intracellular transport of cholesterol and other lipids. Cholesterol NPC intracellular cholesterol transporter 1 Homo sapiens